Development and external validation of prediction models for adverse health outcomes in rheumatoid arthritis : A multinational real-world cohort analysis
Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved..
BACKGROUND: Identification of rheumatoid arthritis (RA) patients at high risk of adverse health outcomes remains a major challenge. We aimed to develop and validate prediction models for a variety of adverse health outcomes in RA patients initiating first-line methotrexate (MTX) monotherapy.
METHODS: Data from 15 claims and electronic health record databases across 9 countries were used. Models were developed and internally validated on Optum® De-identified Clinformatics® Data Mart Database using L1-regularized logistic regression to estimate the risk of adverse health outcomes within 3 months (leukopenia, pancytopenia, infection), 2 years (myocardial infarction (MI) and stroke), and 5 years (cancers [colorectal, breast, uterine] after treatment initiation. Candidate predictors included demographic variables and past medical history. Models were externally validated on all other databases. Performance was assessed using the area under the receiver operator characteristic curve (AUC) and calibration plots.
FINDINGS: Models were developed and internally validated on 21,547 RA patients and externally validated on 131,928 RA patients. Models for serious infection (AUC: internal 0.74, external ranging from 0.62 to 0.83), MI (AUC: internal 0.76, external ranging from 0.56 to 0.82), and stroke (AUC: internal 0.77, external ranging from 0.63 to 0.95), showed good discrimination and adequate calibration. Models for the other outcomes showed modest internal discrimination (AUC < 0.65) and were not externally validated.
INTERPRETATION: We developed and validated prediction models for a variety of adverse health outcomes in RA patients initiating first-line MTX monotherapy. Final models for serious infection, MI, and stroke demonstrated good performance across multiple databases and can be studied for clinical use.
FUNDING: This activity under the European Health Data & Evidence Network (EHDEN) has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 806968. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA.
| Medienart: |
E-Artikel |
|---|
| Erscheinungsjahr: |
2022 |
|---|---|
| Erschienen: |
2022 |
| Enthalten in: |
Zur Gesamtaufnahme - volume:56 |
|---|---|
| Enthalten in: |
Seminars in arthritis and rheumatism - 56(2022) vom: 15. Okt., Seite 152050 |
| Sprache: |
Englisch |
|---|
| Beteiligte Personen: |
Yang, Cynthia [VerfasserIn] |
|---|
| Links: |
|---|
| Themen: |
Antirheumatic Agents |
|---|
| Anmerkungen: |
Date Completed 08.09.2022 Date Revised 22.03.2023 published: Print-Electronic Citation Status MEDLINE |
|---|
| doi: |
10.1016/j.semarthrit.2022.152050 |
|---|
| funding: |
|
|---|---|
| Förderinstitution / Projekttitel: |
|
| PPN (Katalog-ID): |
NLM342496301 |
|---|
| LEADER | 01000naa a22002652 4500 | ||
|---|---|---|---|
| 001 | NLM342496301 | ||
| 003 | DE-627 | ||
| 005 | 20231226014241.0 | ||
| 007 | cr uuu---uuuuu | ||
| 008 | 231226s2022 xx |||||o 00| ||eng c | ||
| 024 | 7 | |a 10.1016/j.semarthrit.2022.152050 |2 doi | |
| 028 | 5 | 2 | |a pubmed24n1141.xml |
| 035 | |a (DE-627)NLM342496301 | ||
| 035 | |a (NLM)35728447 | ||
| 035 | |a (PII)S0049-0172(22)00101-9 | ||
| 040 | |a DE-627 |b ger |c DE-627 |e rakwb | ||
| 041 | |a eng | ||
| 100 | 1 | |a Yang, Cynthia |e verfasserin |4 aut | |
| 245 | 1 | 0 | |a Development and external validation of prediction models for adverse health outcomes in rheumatoid arthritis |b A multinational real-world cohort analysis |
| 264 | 1 | |c 2022 | |
| 336 | |a Text |b txt |2 rdacontent | ||
| 337 | |a ƒaComputermedien |b c |2 rdamedia | ||
| 338 | |a ƒa Online-Ressource |b cr |2 rdacarrier | ||
| 500 | |a Date Completed 08.09.2022 | ||
| 500 | |a Date Revised 22.03.2023 | ||
| 500 | |a published: Print-Electronic | ||
| 500 | |a Citation Status MEDLINE | ||
| 520 | |a Copyright © 2022 The Author(s). Published by Elsevier Inc. All rights reserved. | ||
| 520 | |a BACKGROUND: Identification of rheumatoid arthritis (RA) patients at high risk of adverse health outcomes remains a major challenge. We aimed to develop and validate prediction models for a variety of adverse health outcomes in RA patients initiating first-line methotrexate (MTX) monotherapy | ||
| 520 | |a METHODS: Data from 15 claims and electronic health record databases across 9 countries were used. Models were developed and internally validated on Optum® De-identified Clinformatics® Data Mart Database using L1-regularized logistic regression to estimate the risk of adverse health outcomes within 3 months (leukopenia, pancytopenia, infection), 2 years (myocardial infarction (MI) and stroke), and 5 years (cancers [colorectal, breast, uterine] after treatment initiation. Candidate predictors included demographic variables and past medical history. Models were externally validated on all other databases. Performance was assessed using the area under the receiver operator characteristic curve (AUC) and calibration plots | ||
| 520 | |a FINDINGS: Models were developed and internally validated on 21,547 RA patients and externally validated on 131,928 RA patients. Models for serious infection (AUC: internal 0.74, external ranging from 0.62 to 0.83), MI (AUC: internal 0.76, external ranging from 0.56 to 0.82), and stroke (AUC: internal 0.77, external ranging from 0.63 to 0.95), showed good discrimination and adequate calibration. Models for the other outcomes showed modest internal discrimination (AUC < 0.65) and were not externally validated | ||
| 520 | |a INTERPRETATION: We developed and validated prediction models for a variety of adverse health outcomes in RA patients initiating first-line MTX monotherapy. Final models for serious infection, MI, and stroke demonstrated good performance across multiple databases and can be studied for clinical use | ||
| 520 | |a FUNDING: This activity under the European Health Data & Evidence Network (EHDEN) has received funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 806968. This Joint Undertaking receives support from the European Union's Horizon 2020 research and innovation programme and EFPIA | ||
| 650 | 4 | |a Journal Article | |
| 650 | 4 | |a Research Support, Non-U.S. Gov't | |
| 650 | 4 | |a Cardiovascular diseases | |
| 650 | 4 | |a Infections | |
| 650 | 4 | |a Methotrexate | |
| 650 | 4 | |a Prediction models | |
| 650 | 4 | |a Rheumatoid arthritis | |
| 650 | 7 | |a Antirheumatic Agents |2 NLM | |
| 650 | 7 | |a Methotrexate |2 NLM | |
| 650 | 7 | |a YL5FZ2Y5U1 |2 NLM | |
| 700 | 1 | |a Williams, Ross D |e verfasserin |4 aut | |
| 700 | 1 | |a Swerdel, Joel N |e verfasserin |4 aut | |
| 700 | 1 | |a Almeida, João Rafael |e verfasserin |4 aut | |
| 700 | 1 | |a Brouwer, Emily S |e verfasserin |4 aut | |
| 700 | 1 | |a Burn, Edward |e verfasserin |4 aut | |
| 700 | 1 | |a Carmona, Loreto |e verfasserin |4 aut | |
| 700 | 1 | |a Chatzidionysiou, Katerina |e verfasserin |4 aut | |
| 700 | 1 | |a Duarte-Salles, Talita |e verfasserin |4 aut | |
| 700 | 1 | |a Fakhouri, Walid |e verfasserin |4 aut | |
| 700 | 1 | |a Hottgenroth, Antje |e verfasserin |4 aut | |
| 700 | 1 | |a Jani, Meghna |e verfasserin |4 aut | |
| 700 | 1 | |a Kolde, Raivo |e verfasserin |4 aut | |
| 700 | 1 | |a Kors, Jan A |e verfasserin |4 aut | |
| 700 | 1 | |a Kullamaa, Lembe |e verfasserin |4 aut | |
| 700 | 1 | |a Lane, Jennifer |e verfasserin |4 aut | |
| 700 | 1 | |a Marinier, Karine |e verfasserin |4 aut | |
| 700 | 1 | |a Michel, Alexander |e verfasserin |4 aut | |
| 700 | 1 | |a Stewart, Henry Morgan |e verfasserin |4 aut | |
| 700 | 1 | |a Prats-Uribe, Albert |e verfasserin |4 aut | |
| 700 | 1 | |a Reisberg, Sulev |e verfasserin |4 aut | |
| 700 | 1 | |a Sena, Anthony G |e verfasserin |4 aut | |
| 700 | 1 | |a Torre, Carmen O |e verfasserin |4 aut | |
| 700 | 1 | |a Verhamme, Katia |e verfasserin |4 aut | |
| 700 | 1 | |a Vizcaya, David |e verfasserin |4 aut | |
| 700 | 1 | |a Weaver, James |e verfasserin |4 aut | |
| 700 | 1 | |a Ryan, Patrick |e verfasserin |4 aut | |
| 700 | 1 | |a Prieto-Alhambra, Daniel |e verfasserin |4 aut | |
| 700 | 1 | |a Rijnbeek, Peter R |e verfasserin |4 aut | |
| 773 | 0 | 8 | |i Enthalten in |t Seminars in arthritis and rheumatism |d 1971 |g 56(2022) vom: 15. Okt., Seite 152050 |w (DE-627)NLM000215643 |x 1532-866X |7 nnns |
| 773 | 1 | 8 | |g volume:56 |g year:2022 |g day:15 |g month:10 |g pages:152050 |
| 856 | 4 | 0 | |u http://dx.doi.org/10.1016/j.semarthrit.2022.152050 |3 Volltext |
| 912 | |a GBV_USEFLAG_A | ||
| 912 | |a GBV_NLM | ||
| 951 | |a AR | ||
| 952 | |d 56 |j 2022 |b 15 |c 10 |h 152050 | ||